Aiming for a reduced pediatric Hemophilia Activities List!

Introduction : The pediatric Hemophilia Activities List (pedHAL) assesses self-reported limitations in activities in children and youth with hemophilia. The pedHAL contains 53 items, divided in seven domains. After introducing the pedHAL clinicians and researchers reported on non-informative items. In this explorative field test we aimed to systematically evaluate all items of the pedHAL in Dutch patients with access to early prophylaxis, with the aim to potentially shorten the pedHAL. In addition, we determined change scores over 3 and 5 years to explore the optimal frequency of measurement. Methods: We analyzed longitudinal pedHAL data collected at the annual routine assessment at the Van Creveldkliniek, University Medical Center Utrecht, The Netherlands between October 2010 - June 2017. Patients (4-18 years) with moderate/severe hemophilia A/B and their parents completing ≥1 pedHAL were included. Descriptive analyses were performed per item of the last completed pedHAL per child/parent to determine the distribution of scoring. Change scores were calculated in parents for the sum score for 3 and 5 years follow-up. Results: Eighty-two patients with hemophilia A/B (severe 94%, median 13.1 years [range 5.4-18.0]) were included in this study. In children 22 items were scored as ‘never problems’ in >90% and 18 items were scored as ‘never problems’ in 80-90%. In parents 15 items were scored as ‘never problems’ in >90% and 28 items were scored as ‘never problems’ in 80-90%. The 13 most informative items (<80% ‘never problems’) belonged to the 3 domains Sitting/kneeling/standing, Functions of the legs and Leisure activities and sports. Most items scored >15% as ‘not applicable’ belonged to the domains of Household tasks and Leisure activities and sports. Median (IQR) change sum score after 3 and 5 years follow-up was respectively 0.0 (-2.2;0.1) and 0.0 (-1.1;0.4). Discussion/Conclusion: Although the time to complete the pedHAL is 10-15 minutes, item reduction is expected enhance feasibility of the pedHAL for research and clinical proposes. This explorative analysis suggests that the number of items can be reduced, as several items appear non-informative. Lack of change in scores over 3 and 5 years suggests that annual pedHAL administration is not necessary. However, this study was performed in a single-center sample and therefore needs to be expanded to include patients treated with less intensive regimens before the pedHAL can be shortened.